for people ages 18-65 (full criteria)
at Sacramento, California and other locations
study started
estimated completion
Principal Investigator
by Valentina Medici



The objectives of this clinical trial are to assess, for up to 5 years, the safety, tolerability and pharmacological activity of a single ascending doses of VTX-801, a gene therapy, administered intravenously (IV) to adult patients with Wilson's Disease prior to and following background WD therapy withdrawal.

Official Title

A Phase I/II, Multicenter, Non-randomized, Open Label, Adaptive Design, 5-year Follow-up, Single Dose-escalation Study of VTX-801 in Adult Patients With Wilson's Disease


Wilson's Disease Hepatolenticular Degeneration VTX-801


You can join if…

Open to people ages 18-65

  • Male or female aged between 18 and 65 years
  • Patient diagnosed with WD, as historically established and documented by Leipzig score ≥ 4, as per the 2012 EASL Clinical Practice Guidelines (EASL, 2012) and genetically confirmed
  • Treated for WD according to international recommendations with no current evidence for inadequate treatment (EASL, 2012; Roberts et al, 2008)
  • Stable WD for ≥ 1 year, defined as: (i) No significant change in neurologic examination and in status of mood disorder, if present, as judged by a physician-expert(s) in assessing neurological and psychiatric manifestations of WD and (ii) Stable laboratory parameters used to assess copper metabolism including 24-hour urinary copper, non-ceruloplasmin-bound copper (NCC), as well as liver enzymes, hemoglobin, and white blood cell count

You CAN'T join if...

  • ALT level (mean of 2 measurements ≥ 1-week apart) > ULN or either of the 2 measurements > 1.5 x ULN
  • Total bilirubin > 1.5 x ULN in the absence of proven Gilbert's syndrome; in case of Gilbert's syndrome, direct bilirubin > ULN
  • INR > ULN
  • Any signs of liver cirrhosis decompensation, including gastrointestinal bleed within 6 months (24 weeks) prior to screening/enrolment visit
  • Patient has moderate or severe renal impairment defined as eGFR CKD-EPI < 60 mL/min/1.73 m2, or patient has nephritis or nephrotic syndrome
  • Any history or current evidence of HIV-1, HIV-2, HTLV 1, or HTLV-2 infection
  • Any history or current evidence of hepatitis B infection
  • Any history of hepatitis C infection, unless previous viral RNA assays in two samples, collected at least 6 months apart, are negative
  • Positive QuantiFERON®-TB Gold tuberculosis test result
  • Any concomitant disorder/condition - including hepatic disorders - or treatment possibly interfering with the conduct or evaluation of the study, according to the Investigator
  • Pregnancy or breastfeeding
  • Body Mass Index ≥ 35 kg/m2


  • UC Davis Medical Center accepting new patients
    Sacramento California 95817 United States
  • University of Michigan Health System accepting new patients
    Ann Arbor Michigan 48109 United States

Lead Scientist at UC Davis


accepting new patients
Start Date
Completion Date
Vivet Therapeutics SAS
Sign up for this study
Phase 1/2 Wilson's Disease Research Study
Study Type
Expecting 16 study participants
Last Updated